Tazemetostat Granted Orphan Drug Status for Rare, Aggressive Cancers

Cancer Cells
Cancer Cells

Epizyme announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to tazemetostat for the treatment of malignant rhabdoid tumors (MRTs).

MRTs are rare and aggressive cancers defined by a loss of INI1 protein as measured by immunohistochemistry. In the ovary, MRT is characterized by a loss of the SMARCA4 protein. The Orphan Drug designation applies to both INI1-negative and SMARCA4-negative types. Currently, no target-directed therapy exists for MRTs.

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Epizyme is evaluating tazemetostat in ongoing adult Phase 2, and pediatric Phase 1 studies, in patients with genetically-defined solid tumors, including those with rhabdoid tumors, other INI1-negative tumors, and synovial sarcoma. The company is also investigating tazemetostat for non-Hodgkin's lymphoma in an ongoing Phase 2 study.

Tazemetostat (EPZ-6438) is a first-in-class small molecule inhibitor of EZH2, a histone methyltransferase (HMT) enzyme with oncogenic activity.

For more information visit Epizyme.com.

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